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The use of the non-parametric Restricted Mean Survival Time endpoint (RMST) has grown in popularity as trialists look to analyse time-to-event outcomes without the restrictions of the proportional hazards assumption. In this paper, we…

统计方法学 · 统计学 2023-11-06 Emily Alger , David S. Robertson , Abigail J. Burdon

In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs…

统计方法学 · 统计学 2022-09-07 Xueqi Wang , Elizabeth L. Turner , John S. Preisser , Fan Li

Hybrid studies allow investigators to simultaneously study an intervention effectiveness outcome and an implementation research outcome. In particular, type 2 hybrid studies support research that places equal importance on both outcomes…

统计方法学 · 统计学 2024-11-15 Melody A. Owen , Geoffrey M. Curran , Justin D. Smith , Yacob Tedla , Chao Cheng , Donna Spiegelman

Heterogeneous treatment effect models allow us to compare treatments at subgroup and individual levels, and are of increasing popularity in applications like personalized medicine, advertising, and education. In this talk, we first survey…

统计方法学 · 统计学 2022-01-28 Zijun Gao , Trevor Hastie

This paper addresses one of the most prevalent problems encountered by political scientists working with difference-in-differences (DID) design: missingness in panel data. A common practice for handling missing data, known as complete case…

统计方法学 · 统计学 2024-12-02 Sooahn Shin

Marginal structural models fit via inverse probability of treatment weighting are commonly used to control for confounding when estimating causal effects from observational data. When planning a study that will be analyzed with marginal…

应用统计 · 统计学 2020-03-16 Bonnie E. Shook-Sa , Michael G. Hudgens

Difference-in-differences (DiD) identification relies mainly on a parallel trends assumption about untreated potential outcomes. Researchers often relax this assumption by assuming conditional parallel trends within units with the same…

统计方法学 · 统计学 2026-05-05 Daniela Rodrigues , Laura A. Hatfield

We propose a difference-in-differences (DiD) framework with mediation for possibly multivalued discrete or continuous treatments and mediators, aimed at identifying the direct effect of the treatment on the outcome (net of effects operating…

计量经济学 · 经济学 2026-03-02 Martin Huber , Sarina Joy Oberhänsli

Often both Aggregate Data (AD) studies and Individual Patient Data (IPD) studies are available for specific treatments. Combining these two sources of data could improve the overall meta-analytic estimates of treatment effects. Moreover,…

统计方法学 · 统计学 2021-11-15 Neha Agarwala , Junyong Park , Anindya Roy

Difference-in-differences is based on a parallel trends assumption, which states that changes over time in average potential outcomes are independent of treatment assignment, possibly conditional on covariates. With time-varying treatments,…

统计方法学 · 统计学 2024-06-25 Nicholas Illenberger , Iván Díaz , Audrey Renson

This paper studies the estimation of linear panel data models with interactive fixed effects, where one dimension of the panel, typically time, may be fixed. To this end, a novel transformation is introduced that reduces the model to a…

计量经济学 · 经济学 2021-10-13 Ayden Higgins

There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map…

统计方法学 · 统计学 2022-02-22 Eric J. Rose , Erica E. M. Moodie , Susan Shortreed

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

统计方法学 · 统计学 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

In this work we provide a couple of contributions to the analysis of longitudinal data collected by smartphones in mobile health applications. First, we propose a novel statistical approach to disentangle personalized treatment and…

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…

统计方法学 · 统计学 2025-10-28 Xi Fang , Zhiqiang Cao , Fan Li

We address the problem of estimating heterogeneous treatment effects in panel data, adopting the popular Difference-in-Differences (DiD) framework under the conditional parallel trends assumption. We propose a novel doubly robust…

机器学习 · 统计学 2025-04-29 Hui Lan , Haoge Chang , Eleanor Dillon , Vasilis Syrgkanis

Consider a general setting in which data on an outcome is collected in two `groups' at two time periods, with certain group-periods deemed `treated' and others `untreated'. A special case is the canonical Difference-in-Differences (DiD)…

统计方法学 · 统计学 2025-09-15 Zach Shahn , Laura Hatfield

Sequential, multiple assignment, randomized trial (SMART) designs have become increasingly popular in the field of precision medicine by providing a means for comparing sequences of treatments tailored to the individual patient, i.e.,…

应用统计 · 统计学 2018-04-13 William J. Artman , Inbal Nahum-Shani , Tianshuang Wu , James R. McKay , Ashkan Ertefaie

Difference-in-differences (DiD) is the most popular observational causal inference method in health policy, employed to evaluate the real-world impact of policies and programs. To estimate treatment effects, DiD relies on the "parallel…

应用统计 · 统计学 2024-08-09 Shuo Feng , Ishani Ganguli , Youjin Lee , John Poe , Andrew Ryan , Alyssa Bilinski

We present an aggregation scheme that increases power in randomized controlled trials and quasi-experiments when the intervention possesses a robust and well-articulated theory of change. Longitudinal data analyzing interventions often…

统计方法学 · 统计学 2022-07-27 Timothy Lycurgus , Ben B. Hansen